you’re not alone on your myelofibrosis journey

VO: Vonjo or pacritinib capsules are a prescription medicine used to treat adults with certain types of myelofibrosis, or MF, who have a platelet count below 50,000 per microliter. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may depend on proof and description of clinical benefit in a confirmatory trial or confirmatory trials. It is not known if Vonjo is safe and works in children.

Jennine: My name is Jennine. I'm 64 years old and I was diagnosed with myelofibrosis in 2021. I retired in 2018, and I worked as a medical assistant for a podiatrist for 37 years. I live with my husband and our youngest son. All together we have three sons and some very beautiful grandchildren. I love cooking, baking, walking the dogs. Life does have its challenges, but I do try to stay active and maintain a healthy lifestyle.

I was diagnosed with essential thrombocythemia, or ET, in 2011. My primary care physician noticed some abnormalities in my routine bloodwork, so she sent me to a hematologist. At that time, I wasn't feeling much different and wasn't experiencing any noticeable symptoms. I didn't know what to make of it. We then monitored things and I continued on with my life.

Then over the next few years, in 2016, it progressed to Polycythemia Vera or PV, and that's when the symptoms slowly started to creep in. I started getting more fatigued. I pushed through the long days of the clinic thinking I was just working too hard, or getting older, or taking care of my three sons.

Eventually, though, things were getting progressively worse. The fatigue was horrible. I had massive bruises on my arms, legs and even on my torso. I looked like I was beaten up. My spleen became so enlarged and even at the worst part, it felt as though it was half the size of my torso. The hematologist I was seeing retired, and he told me that I need more specialized care. Then when I started seeing Dr Rossetti, I was seeing him every few weeks.

Dr Rossetti: Hello. My name is Dr. James Rossetti. I'm a hematologist in Pittsburgh, Pennsylvania, specializing in myeloid malignancies, including myelofibrosis.

We see many patients with myeloproliferative neoplasms or MPN’s, perhaps second opinions or involvement in more complex cases. I've been very fortunate to walk alongside many patients suffering with these chronic illnesses, but Jennine's story is one that really stands out to me and one I will never forget.

When Jennine first came to me in the summer of 2021, her myelofibrosis was already at a high-risk stage. Several cell lines were already low. She was feeling poorly, very tired, and had a poor appetite. Her spleen was significantly enlarged. She had already failed a prior therapy and had to discontinue another due to troubling side effects.

At that time, we were looking at one of two options moving forward. A trial drug called Vonjo or a bone marrow transplant. I was very honest with Jennine stating that I was skeptical if any drug would help her disease for a long period of time. I told her that the likelihood is that we would be headed to bone marrow transplant fairly quickly.

VO: VONJO (pacritinib) capsules can cause the following serious side effects:

• Severe bleeding, which can be serious, and in some cases may lead to death. Stop taking VONJO and tell your healthcare provider right away if you develop any unusual bleeding, bruising, and fever. Get medical help right away for any bleeding that you cannot stop.
• Diarrhea. Diarrhea is common with VONJO, but can be severe and cause loss of too much body fluid (dehydration), which may cause kidney problems. Tell your healthcare provider if you have diarrhea and follow instructions for what to do if you get any symptoms of diarrhea (including taking an antidiarrheal). Drink plenty of fluids to help prevent dehydration. Urgently seek emergency medical attention if diarrhea becomes severe.
• Worsening low platelet counts.
• Changes in the electrical activity of your heart called QTc prolongation which can cause irregular heartbeats that can be life-threatening. Tell your healthcare provider right away if you feel dizzy, lightheaded, or faint.

Get emergency help right away if you have any symptoms of a heart attack or stroke during treatment with VONJO. You may be at increased risk if you have cardiovascular risk factors, smoke or smoked in the past.

Tell your healthcare provider right away if you have any signs and symptoms of blood clots during treatment with VONJO.

There is a possible increased risk of new (secondary) cancers with Vonjo. You may be at increased risk if you smoke or smoked in the past.

People who take VONJO may develop serious infections. If you have a serious infection, your healthcare provider may not start you on VONJO until your infection is gone. Tell your healthcare provider right away if you develop any symptoms of infection.

Please see additional important safety information at the end of this video.

Dr Rossetti: That's the nature of myelofibrosis. It truly is a step-by-step journey. Jennine was feeling very poorly physically when she first came to me, but what really stood out to me was how mentally present she was. Her openness and courage laid a foundation for trust that followed.

Jennine: Early in our conversations, he told me about Vonjo. He said, I'd like you to try this medication. It's a clinical trial drug, but there has been some promising data with it. He took the time to explain how it worked, what the early research was showing and what it could mean for someone in my situation.

Dr Rossetti: I explained Vonjo's trial design to Jennine. In a six-month clinical study, some people received 200 mg of Vonjo twice a day and some people receive best available therapy or BAT, which included ruxolitinib, hydroxyurea, and monitoring without treatment. For people with low platelet counts, less than 50,000 platelets per microliter, 30% of people on Vonjo had their spleen get at least 35% smaller compared to 3% of people on BAT. The most common side effects that occurred in more than 20% of people in the study were diarrhea, nausea, low platelet counts or thrombocytopenia, or low red blood cell counts, anemia and swelling of ankles, legs and feet.

Jennine: I remember asking him something very personal. I said, if this was your sister or your mother, what would you do? And without any hesitation, he said, this is exactly what I would do. I didn't know what to expect. I was really scared, but I knew I couldn't go on like this anymore. When I sat down with Dr Rossetti, he didn't just list the facts, he explained things in a way that felt grounded and honest. He gave me hope and something real to hold on to.

So I started Vonjo in August 2021. From the beginning, I was monitored closely, regular bloodwork, check ins with my care team and lab works every week. It was slow going in the beginning, but over time I began to notice little shifts. The bruising started to fade, my appetite picked up. I didn't feel as fragile. The size of my spleen reduced. The fatigue was still there, but it wasn't crushing anymore. I could get through the mornings without needing to lie down. I started walking the dogs again. I could cook and bake and do small errands, and I wasn't just surviving the day, I was participating in it. This is my experience with Vonjo other people may react differently.

Dr Rossetti: With Jennine, we have seen very encouraging clinical results. Many of the key parameters we follow, have shown improvement, her spleen size has reduced, her appetite has returned, her energy has increased, and we continue to monitor her blood counts.

But it truly wasn't just about the numbers. It was about Jennine telling me I walked the dog today or I played with the grandchildren.

Jennine: I still do get tired in the afternoons, but that part hasn't completely gone away. But now I know how to manage it. I rest when I need to. I pace myself, and I don't push too hard.

Living with the condition, like myelofibrosis, comes with a lot of unknowns. For me, it's important that my doctor sees me as a whole person, not just a diagnosis. I knew I needed someone who would take the time to listen, explain things clearly and involve me in every decision.

From the very first appointment with Dr. Rossetti, I felt something different. He sat down like he wasn't in a rush and asked how I was doing. Not just physically, but emotionally. That doesn't happen often, and it really stayed with me.  It's not just about numbers from a lab result. It's about conversations, trust, and knowing your doctor understands what matters in your life. He's not only an expert, but I know he has my best interests at heart. I honestly can't say enough good things about him. He remembers things I told him months earlier, asks about my family and generally cares. I even get a hug from him when he walks in the room. That kind of human connection makes all the difference. It reminds you that you're not just a patient, you're a person. And he sees that. That's how we manage my myelofibrosis by talking about the whole picture together.

Dr Rossetti: Jennine is the type of person you remember long after they leave the clinic. Jennine is deeply invested in her care, she comes prepared to office visits with notes and articles and written questions. At our first visit, she admitted that she didn't have a deep knowledge of the disease. Jennine brought her daughter-in-law, who is a nurse, to her first appointment. When you're talking about something as serious as transplant or clinical drugs, it's important to have someone with you. Someone that ensures that you both heard the same thing, or someone that can just help you remember what was said. Her daughter-in-law asked wonderful questions and continues to be an advocate for her today. This type of support is so important along your journey and cannot be overstated.

Jennine's experience highlights that treating patients with myelofibrosis is about much more than just managing a disease. Her story is an example of what can happen when a patient finds the right treatment team. A team that works together. Your doctor should make you feel seen and see you as more than a diagnosis, but rather as a whole person. Jennine and I have developed a partnership based on trust, empathy and transparency. It's a model for how healthcare should feel.

As I always tell patients, we want you alive and we want you living. Jennine has gotten to that place. She's feeling much more positive about where she is. You can see it in her face and in her demeanor. I couldn't be prouder of Jennine and more honored to be a part of her journey.

Jennine: When I think about where I was and where I am today, it feels like quite the journey.

My advice? Don't wait. Advocate for yourself. Ask questions, and don't be afraid to speak up. And most important of all, find a doctor who respects you and supports you. It's that type of relationship that can make all the difference. You need someone you feel safe with, who listens to you and fights for you. That's what I have in Dr. Rossetti. There is help out there. Vonjo helped me get there, and Dr. Rossetti helped me every step of the way.

VO:

Important Safety Information

Do not use VONJO if you are taking other medications that are strong CYP3A4 inhibitors or inducers.

What are the serious side effects of VONJO?

Bleeding. VONJO can cause severe bleeding, which can be serious and, in some cases, may lead to death.

• Stop taking VONJO and tell your healthcare provider right away if you develop any of these symptoms: unusual bleeding, bruising, and fever. Get medical help right away for any bleeding that you cannot stop
• You will need to stop taking VONJO 7 days before any planned surgery or invasive procedure (such as a heart catheterization, stent placement in a coronary artery in your heart, or a procedure for varicose veins). Your healthcare provider should tell you when you can start taking VONJO again

Diarrhea. Diarrhea is common with VONJO, but can be severe and cause loss of too much body fluid (dehydration), which may cause kidney problems. Tell your healthcare provider if you have diarrhea and follow instructions for what to do if you get any symptoms of diarrhea (including taking an antidiarrheal). Drink plenty of fluids to help prevent dehydration. Urgently seek emergency medical attention if diarrhea becomes severe.

Worsening low platelet counts.

Changes in the electrical activity of your heart called QTc prolongation. QTc prolongation can cause irregular heartbeats that can be life-threatening. Tell your healthcare provider right away if you feel dizzy, lightheaded, or faint.

Increased risk of major cardiovascular events such as heart attack, stroke, or death in people have happened, especially in those who have cardiovascular risk factors and who are current or past smokers taking another Janus associated kinase (JAK) inhibitor to treat rheumatoid arthritis.

Get emergency help right away if you have any symptoms of a heart attack or stroke during treatment with VONJO, including: discomfort in the center of your chest that lasts for more than a few minutes, or that goes away and comes back; severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw; pain or discomfort in your arms, back, neck, jaw, or stomach; shortness of breath with or without chest discomfort; breaking out in a cold sweat; nausea or vomiting; feeling lightheaded; weakness in one part or on one side of your body; or slurred speech.

Increased risk of blood clots. Blood clots in the veins of your legs (deep vein thrombosis, DVT) or lungs (pulmonary embolism, PE) have happened in some people taking another JAK inhibitor for rheumatoid arthritis and may be life-threatening.

Tell your healthcare provider right away if you have any signs and symptoms of blood clots during treatment with VONJO, including: swelling, pain, or tenderness in one or both legs; sudden, unexplained chest pain; or shortness of breath/difficulty breathing.

Possible increased risk of new (secondary) cancers. People who take another JAK inhibitor for rheumatoid arthritis have an increased risk of new (secondary) cancers, including lymphoma and other cancers, except non-melanoma skin cancer. The risk of new cancers is further increased in people who smoke or have smoked in the past.

Risk of infection. People who have certain blood cancers and take another JAK inhibitor have an increased risk of serious infections. People who take VONJO may develop serious infections, including bacterial, mycobacterial, fungal, and viral infections. If you have a serious infection, your healthcare provider may not start you on VONJO until your infection is gone. Your healthcare provider will monitor you and treat you for any infections that you get during treatment with VONJO.

Tell your healthcare provider right away if you develop any of the following symptoms of infection: chills, aches, fever, nausea, vomiting, weakness, painful skin rash, or blisters.

Tell your healthcare provider about all the medicines you take, including prescription and over-the counter medicines, vitamins, and herbal supplements and remedies. Taking VONJO with certain other medicines may affect the amount of VONJO or the other medicines in your blood and may increase your risk of side effects or affect how well VONJO works.

Especially tell your healthcare provider if you take hormonal contraceptives (birth control). Hormonal birth control methods, except for intrauterine systems containing levonorgestrel, may not work during treatment with VONJO and for 30 days after your last dose. Talk to your healthcare provider about birth control methods that may be right for you during treatment with VONJO.

Your healthcare provider may change your dose or how often you take VONJO, temporarily stop, or permanently stop treatment with VONJO if you have certain side effects.

What should I tell my healthcare provider before starting VONJO?

• Have active bleeding, have had severe bleeding, or plan to have surgery or noninvasive procedures.
• You should stop taking VONJO 7 days before any planned surgery or invasive procedures
• Have diarrhea or commonly have loose stools
• Have had a blood clot, heart attack, other heart problems, or stroke
• Have a history of low blood levels of potassium. It is important that you get blood tests done during
• treatment with VONJO to monitor your body salts (electrolytes)
• Smoke or were a smoker in the past
• Have had any other cancers
• Have an infection
• Have nausea or vomiting
• Have liver or kidney problems
• Are pregnant or plan to become pregnant. It is not known if VONJO will harm your unborn baby
• Are breastfeeding or plan to breastfeed. It is not known if VONJO passes into your breast milk. You should not breastfeed during treatment and for 2 weeks after your last dose of VONJO. Talk to your healthcare provider about the best way to feed your baby during this time

What are the most common side effects with VONJO?

Low platelet count (thrombocytopenia), nausea, vomiting, low red blood cell count (anemia), and swelling of your ankles, legs, and feet.

Your healthcare provider will do blood tests and an electrocardiogram (ECG) before you start treatment with VONJO and as needed during treatment to check for side effects.

VONJO may affect fertility in males. You may have problems fathering a child. Talk to your healthcare provider if this is a concern for you.

These are not all of the possible side effects with VONJO.

If you suspect that you have experienced a side effect, call your healthcare provider. You may also report side effects to the US Food and Drug Administration (FDA) at 1-800-FDA-1088.

Please see the full Prescribing Information for VONJO at www.vonjo.com.